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One of the most common sphingolipidosis, Gaucher disease (GD) remains rare till date. A case report of a 56-year-old patient diagnosed with GD is presented herein. Her sister was known to have Gaucher disease. Her personal medical record consisted of splenectomy, anemia, recurrent infections, and bone lesions at a young age. Taking into consideration her personal and familial history, the clinical and paraclinical examinations, she was diagnosed with Gaucher disease which was confirmed with enzyme and gene testing. Upon introduction of specific enzyme replacement treatment for Gaucher patients,much evidence demonstrated the substantial improvement of hematological and visceral parameters. However, it has been observed that the bone tissue does not respond equally to the treatment.
• The physician should always investigate the splenomegaly of unknown etiology before deciding to do a splenectomy
• Bone lesions in Gaucher disease are sometimes irreversible, hence the importance of early diagnosis of this rare disease
Gaucher disease; Bone lesion; Splenomeagly.
GD: Gaucher disease; Hb: Hemoglobin; MRI: Magnetic resonance imaging; ERT: Enzyme replacement therapy.
It is difficult for the elderly, those with complications, and those who live in remote areas to visit the hospital, and as a result, there are limits on the drugs they are able to use. It is therefore effective to prescribe such patients oral medications that have few adverse effects and in regimens that require few hospital visits. Clarithromycin can induce cell death by autophagy and it has a direct antitumor effect. There have been reports of the outcomes of Lenalidomide and Dexamethasone therapy with Clarithromycin which is administered orally and is safe on multiple myeloma. However, in Japan, there have been few studies. Here, we report on Clarithromycin, Lenalidomide and Dexamethasone therapy in our hospital.
We analyzed 7 patients with relapsed refractory or refractory multiple myeloma who were treated at this hospital between January 2012 and December 2014. The Clarithromycin, Lenalidomide and Dexamethasone therapy were administered in a 28-day cycle as follows: Clarithromycin 400 mg/day for 28-days, Lenalidomide 15 mg/day for 21-days, and Dexamethasone was administered in a dose of 20 mg once per week. The response criteria used were standard International Myeloma Working Group (IMWG) Uniform Response Criteria. and adverse events were graded according to the national cancer institute-common terminology criteria for adverse events (NCI-CTCAE) Ver. 4. Statistical analysis was performed using Easy R (EZR).
The response to Clarithromycin, Lenalidomide and Dexamethasone therapy were selective catalytic reduction (sCR) in 2 patients, CR in 1 patient, per rectum (PR) in 3 patients, and standard deviation (SD) in 1 patient. Response rates of PR or better were observed in 86% of the patients. Duration of response was median 316-days (range, 160-522-days). Median oculus sinister (OS) period was 1,907 days. Median OS following discontinuation of the study was 1,385 days. Hematological adverse events were G1-2 anemia in 3 patients and G3-4 anemia in 1 patient. G1-2 thrombocytopenia was observed in 1 patient and G3-4 thrombocytopenia was observed in 1 patient. Leukopenia of G1-2 was observed in 6 patients but G3 was not observed. Non-hematological adverse events were G1-2 liver disorder in 6 patients, G1-2 skin rash in 3 patients, and G1-2 constipation in 2 patients. G4 adverse events were fainting and duodenal ulcer in 1 patient each.
Clarithromycin, Lenalidomide and Dexamethasone can be safely and effectively administered in the relapsed refractory multiple myeloma
Multiple myeloma; Bird; Clarithromycin, lethal dose (Ld).
Department of vascular Medicine
Peking University Shougang Hospital
NO.9 Jinyuanzhuang Road
Shijingshan District, 100144, Beijing China
Director of Medicine
Department of Medicine
Baruch Padeh Poria Medical Center affiliated to the Faculty of Medicine
Ramat Gan, 5290002, Israel